N Engl J Med September 2024 PMID 39321362

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.

Adam Cuker, Kaan Kavakli, Laurent Frenzel, Jiaan-Der Wang, Jan Astermark, Monica H Cerqueira, Alfonso Iorio, Olga Katsarou-Fasouli, Robert Klamroth, Amy D Shapiro, Cédric Hermans, Akira Ishiguro, Andrew D Leavitt, Johannes B Oldenburg, Margareth C Ozelo

Mentions

129
Peak: 20 in Sep 2024
across 5 sources since Sep 2024

Extended reach

2,076,946
potential audience

Total likes

174

Video views

321

Mentions per month

Sep 2024 – Nov 2024

Where it's discussed

  • News72 articles
  • X / Twitter46 posts
  • Blogs6 articles
  • Facebook3 posts
  • YouTube2 videos

AI synopsis

What people are saying

Overall, the discussions demonstrate strong consensus on the therapy's promise, with comments citing specific trial outcomes such as a 71% reduction in bleeding rates and factor IX activity levels of around 27%, while some posts debate costs and logistical challenges, reflecting both excitement and critical analysis.

AgreementModerate agreement

Most discussions recognize the positive outcomes of the gene therapy, emphasizing significant reductions in bleeding and support for the research findings.

InterestHigh interest

Posts show high interest, with many referencing trial results, long-term benefits, and clinical implications, indicating engagement with the topic.

EngagementModerate engagement

Some discussions delve into specifics like factor IX levels and side effects, suggesting moderate depth of engagement, though a few comments are more superficial.

ImpactHigh impact

The consensus highlights the potential of this therapy to transform treatment for hemophilia B, stressing its significance as a safer and less burdensome alternative.

Abstract in brief

  • Fidanacogene elaparvovec is a genetic therapy for hemophilia B that showed promising results in a phase 1-2a study, maintaining high levels of factor IX activity.*
  • In a phase 3 study involving men aged 18 to 65 with severe hemophilia B, the therapy led to a significant 71% reduction in bleeding episodes over 15 months compared to standard prophylactic treatment.*
  • Despite some participants needing glucocorticoids for side effects, the therapy was generally safe, with no serious adverse infusion events reported.*

Top posts

Most liked on X and Bluesky
Samuel Hume
@DrSamuelBHume · X

2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic infusions of factor IX This trial found that a wildtype factor IX gene therapy reduced bleeding - and the burden of infusions https://t.co/exkrInT3BF https://t.co/m5hFS8fNVc

Sep 30, 2024 · 75 likes · 10 shares · 28,783 followers View post →
Michael Makris
@ProfMakris · X

The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11. 15 month mean IX was 27% (median 23%). 62% received steroids! Paper is behind a paywall. https://t.co/v8TYlsoJGr https://t.co/lHy9XmVNV2

Sep 29, 2024 · 23 likes · 6 shares · 13,931 followers View post →
NEJM
@NEJM · X

Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #Hematology https://t.co/l6xJdTcrhF

Oct 2, 2024 · 18 likes · 4 shares · 935,606 followers View post →
NEJM
@NEJM · X

In participants with hemophilia B, fidanacogene elaparvovec was superior to factor IX prophylaxis, with a lower bleeding rate and stable factor IX expression. Read the full BENEGENE-2 trial results: https://t.co/Lg0GW3rzqZ https://t.co/c3kfs1x2G6

Sep 25, 2024 · 10 likes · 2 shares · 935,606 followers View post →
Justin Morgenstern
@First10EM · X

The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have have some learning to do for future edits (the AI edit is rather jumpy), but it's a start #FOAMed https://t.co/3SAey1VP3e

Nov 17, 2024 · 10 likes · 4 shares · 16,963 followers View post →
flora peyvandi
@flora_peyvandi · X

Gene Therapy with #Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/IEnUKpeQA6

Sep 29, 2024 · 6 likes · 1 share · 2,373 followers View post →

All mentions

Girish Kamat
@kamathemat · X

Phase 3 open-label study, fidanacogene elaparvovec, a gene therapy for hemophilia B, was evaluated in 45 men with severe hemophilia B. The treatment significantly reduced the annualized bleeding rate by 71%. https://t.co/hjTAdGQrr8 Supported by: https://t.co/OwnaMKirrE

Nov 28, 2024 · 1 followers View post →
Kane Guthrie
@kane_guthrie · X

RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…

Nov 17, 2024 · 4 shares · 2,178 followers View post →
Zahir Basrai, MD
@nuMosemed · X

RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…

Nov 17, 2024 · 4 shares · 2,229 followers View post →
Casey Parker
@broomedocs · X

RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…

Nov 17, 2024 · 4 shares · 8,039 followers View post →
Jule R O G Santos
@julesantosER · X

RT @First10EM: The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have…

Nov 17, 2024 · 4 shares · 16,372 followers View post →
Justin Morgenstern
@First10EM · X

The @broomedocs podcast up in video format, although why anyone would want to see us (let alone hear us) is beyond me I have have some learning to do for future edits (the AI edit is rather jumpy), but it's a start #FOAMed https://t.co/3SAey1VP3e

Nov 17, 2024 · 10 likes · 4 shares · 16,963 followers View post →
Ruah Alyamany | روى اليماني
@Ruah_Mahmoud · X

Promising results from the Fidanacogene Elaparvovec gene therapy trial in Hemophilia B suggest potential long-term cost-effectiveness over standard treatments, despite current concerns about high costs. https://t.co/4Xn1i3obcB

Nov 5, 2024 · 1,263 followers View post →
Muhteşem Erol Yayla
@ErolYayla13 · X

Hemofili B'de gen tedavisi yapıldı. Virus vektörü aracılığıyla (Fidanacogene Elaparvovec) gen tedavisi Hemofili B tedavisinde etkili. Bu Hemofili B için kullanılan 2. etkili gen tedavisi oluyor. https://t.co/MW8zt9SBBp

Nov 3, 2024 · 31 followers View post →
Muhteşem Erol Yayla
@ErolYayla13 · X

Hemofili B'de gen tedavisi yapıldı. Virus vektörü aracılığıyla (Fidanacogene Elaparvovec) gen tedavisi Hemofili B tedavisinde etkili. Bu Hemofili B için kullanılan 2. etkili gen tedavisi oluyor. https://t.co/nIAEgfgUua

Nov 3, 2024 · 31 followers View post →
Javier M Cortes MD
@cor38962 · X

RT @wfhemophilia: A study of fidanacogene elaparvovec #GeneTherapy for #hemophilia B demonstrated a 71% reduction in annualized bleeding ra…

Nov 1, 2024 · 3 shares · 6 followers View post →
WFH
@wfhemophilia · X

A study of fidanacogene elaparvovec #GeneTherapy for #hemophilia B demonstrated a 71% reduction in annualized bleeding rates & factor IX levels of 26.9% at 15months following infusion. 16% of the 46 participants reported serious adverse events. Read more➡️ https://t.co/ToXfQl5Hk3 https://t.co/0a6HLs6Nj1

Nov 1, 2024 · 5 likes · 3 shares · 7,595 followers View post →
Pfizer Medical
@PfizerMed · X

View a new hemophilia publication in the NEJM that describes the prospective outcomes of gene therapy as a therapeutic approach for adults diagnosed with #HemophiliaB. Click the link below to learn more: https://t.co/JgEolPSCwi https://t.co/wMybxUyZCN

Oct 24, 2024 · 1 like · 6,961 followers View post →
Richard Buka
@richardbuka · X

In @NEJM, before/after trial of gene therapy for haem B using fidanacogene elaparvovec. n=45, 188 ineligible due to AAV neutralising Abs. Patients had to have been on >6 m factor prophylaxis. Mean FIX level at 15m: 27% & annualised bleeding rate 4.4 ➡️ 1.3 https://t.co/YG0OKmMKaI https://t.co/Nd2fjE7x24

Oct 12, 2024 · 1 like · 1,420 followers View post →
2 Minute Medicine®
@2minmed · X

RT @2minmed: #VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis https://t.c…

Oct 10, 2024 · 1 share · 5,403 followers View post →
Tony McChrystal
@tonymac5 · X

@robertopaulos85 @HorseRenoir83 @HeyNurseKat @_johnbye @Jimmy78591 Yes mate. Virology is certainly on its last legs. You'd best go and tell the people currently using viruses therapeutically. I'm sure they'll bow to the superiority of a random Internet tit with Del Boy as his profile picture.

Oct 9, 2024 · 1 like · 838 followers View post →
2 Minute Medicine®
@2minmed · X

#VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis https://t.co/0WG37Lhplq #StudyGraphics #factorix https://t.co/DtlvWFg6Fg

Oct 9, 2024 · 1 share · 5,403 followers View post →
the paper link
@the_paper_link · X

Trend up+6: Cuker A, ..., Benegene-2 Trial Investigators (N Engl J Med) __Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B__ https://t.co/mvOnPzlQCB

Oct 4, 2024 · 139 followers View post →
Manoj Mayogi Mishra
@mayogisense · X

RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…

Oct 2, 2024 · 4 shares · 573 followers View post →
Cherry Swift
@cherryswift · X

RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…

Oct 2, 2024 · 4 shares · 140 followers View post →
Tom Hsiung
@TomHsiung · X

RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…

Oct 2, 2024 · 4 shares · 463 followers View post →
Ezeonufo Chukwunonso
@Ezeonufo · X

RT @NEJM: Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #…

Oct 2, 2024 · 4 shares · 1,365 followers View post →
NEJM
@NEJM · X

Original Article: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B (BENEGENE-2) https://t.co/Lg0GW3rzqZ #Hematology https://t.co/l6xJdTcrhF

Oct 2, 2024 · 18 likes · 4 shares · 935,606 followers View post →
ISMAIL
@iamaniku · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Oct 2, 2024 · 10 shares · 2,035 followers View post →
CK
@CaseyganC · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Oct 1, 2024 · 10 shares · 1,295 followers View post →
Angelique Trusler
@AngeliquePhd · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Oct 1, 2024 · 10 shares · 587 followers View post →
Debra Adams | #NotGoingBack
@teco22222 · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Oct 1, 2024 · 10 shares · 2,157 followers View post →
Richard Allen
@AHA1R · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Oct 1, 2024 · 10 shares · 5,680 followers View post →
Seth Corey, MD MPH
@sethcorey · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Sep 30, 2024 · 10 shares · 1,130 followers View post →
CopperKettle
@Artyom_Yekat · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Sep 30, 2024 · 10 shares · 379 followers View post →
@gen0m1cs
@gen0m1cs · X

RT @SamuelBHume: 2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic inf…

Sep 30, 2024 · 10 shares · 28,152 followers View post →
Samuel Hume
@DrSamuelBHume · X

2. Haemophilia B, caused by mutations in factor IX, causes bleeding - it's currently treated with regular prophylactic infusions of factor IX This trial found that a wildtype factor IX gene therapy reduced bleeding - and the burden of infusions https://t.co/exkrInT3BF https://t.co/m5hFS8fNVc

Sep 30, 2024 · 75 likes · 10 shares · 28,783 followers View post →
Abstream
@abstreamme · X

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B #science #publication #research #publications https://t.co/3rJT836r80

Sep 30, 2024 · 788 followers View post →
Alfonso Flauto
@fonzo685 · X

RT @flora_peyvandi: Gene Therapy with #Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/IEnUKpeQA6

Sep 30, 2024 · 1 share · 328 followers View post →
Mostafa Faisal
@MostafaFaisal14 · X

RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…

Sep 29, 2024 · 6 shares · 1,050 followers View post →
Faustino García Candel
@FaustinoGCandel · X

RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…

Sep 29, 2024 · 6 shares · 22 followers View post →
flora peyvandi
@flora_peyvandi · X

Gene Therapy with #Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/IEnUKpeQA6

Sep 29, 2024 · 6 likes · 1 share · 2,373 followers View post →
Ruah Alyamany | روى اليماني
@Ruah_Mahmoud · X

RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…

Sep 29, 2024 · 6 shares · 1,263 followers View post →
Andrés Vásconez, MD, FAAP.
@wavasconez · X

RT @ProfMakris: The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11…

Sep 29, 2024 · 6 shares · 429 followers View post →
Michael Makris
@ProfMakris · X

The Pfizer #hemophilia B gene therapy 15 month results are in the current issue of the @NEJM. This is AAV Padua IX at 5e11. 15 month mean IX was 27% (median 23%). 62% received steroids! Paper is behind a paywall. https://t.co/v8TYlsoJGr https://t.co/lHy9XmVNV2

Sep 29, 2024 · 23 likes · 6 shares · 13,931 followers View post →
Dirk Haussecker
@RNAiAnalyst · X

Hemophilia B gene therapy paper on $PFE's Fidanacogene Elaparvovec published in @nejm: https://t.co/5sFR8fYQhp Challenges remaining for field (in order of importance): 1) huge inter-patient variability in Factor IX expression 2) 56% of patients were administered prolonged https://t.co/6fOqHCcOIe

Sep 27, 2024 · 2 likes · 20,236 followers View post →
Frank Stassen
@StassenFrank · X

Het moge duidelijk zijn dat deze gentherapie veel minder belastend is voor patienten dan de huidige standaard, die regelmatig infusies vereist. Voor velen betekent deze nieuwe therapie een leven zonder voortdurende zorg. De hele studie is hier te lezen https://t.co/YFMikMRMfN

Sep 26, 2024 · 6 likes · 1,525 followers View post →
Manuel Muro
@manuelmuro5 · X

Fidanacogene elaparvovec, un vector de terapia génica del virus adenoasociado (AAV) para la hemofilia B que contiene una variante del factor IX humano de alta actividad (FIX-R338L/FIX-Padua), se asoció con una actividad sostenida del factor IX en fase 1-2a https://t.co/o2LcNsnngw

Sep 26, 2024 · 2 likes · 11,197 followers View post →
Dennis Lal
@LalDennis · X

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/Ce4Md5mJYb https://t.co/HbHt2BpXHq

Sep 25, 2024 · 3,309 followers View post →
Phage Papers
@Phage_Papers · X

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B https://t.co/5zpmQWhaGl

Sep 25, 2024 · 647 followers View post →
محمد القاضي
@MohammadAlqady · X

RT @NEJM: In participants with hemophilia B, fidanacogene elaparvovec was superior to factor IX prophylaxis, with a lower bleeding rate and…

Sep 25, 2024 · 2 shares · 357 followers View post →
NEJM
@NEJM · X

In participants with hemophilia B, fidanacogene elaparvovec was superior to factor IX prophylaxis, with a lower bleeding rate and stable factor IX expression. Read the full BENEGENE-2 trial results: https://t.co/Lg0GW3rzqZ https://t.co/c3kfs1x2G6

Sep 25, 2024 · 10 likes · 2 shares · 935,606 followers View post →